FDA moves Sage’s post­par­tum de­pres­sion drug brex­anolone in­to reg­u­la­tors’ busy pri­or­i­ty lane

The FDA’s pri­or­i­ty re­view path­way is get­ting a work­out. 

Reg­u­la­tors agreed to give Sage Ther­a­peu­tics $SAGE a quick 6-month re­view for IV brex­anolone (SAGE-547) — the FDA’s third pri­or­i­ty move in two days — which it hopes will go on to be­come its first mar­ket­ed drug. And this one could be head­ed to a huge mar­ket, if the FDA ap­proves it for post­par­tum de­pres­sion, a con­di­tion that af­flicts a sig­nif­i­cant mi­nor­i­ty of women af­ter giv­ing birth.

The tar­get date for Sage’s drug is De­cem­ber 19, but first the FDA will put the drug through an ex­pert pan­el as­sess­ment with a pub­lic in­spec­tion of an in­ter­nal re­view on the ta­ble.

Sage has al­ready won a break­through ther­a­py des­ig­na­tion for their drug, which could of­fer a big leg up in mar­ket­ing if it’s OK’d. And that’s not a slam dunk.

While Sage scored a pos­i­tive out­come against PPD in two rel­a­tive­ly small Phase III stud­ies, their drug — pro­vid­ed through an in­fu­sion that takes 60 hours to com­plete — didn’t do as well as it did in in Phase II, with the big gap that sep­a­rat­ed the drug and place­bo arms nar­row­ing sig­nif­i­cant­ly.

The main goal of both stud­ies was a sig­nif­i­cant re­duc­tion in de­pres­sion scores 60 hours af­ter treat­ment. And on that lev­el the drug scored a 17.7-point mean re­duc­tion for the high dose and a 19.9-point im­prove­ment for the low dose in the first study for se­vere PPD com­pared to 14 points in the place­bo arm. In study two there was a 14.2-point vs 12-point dif­fer­ence in the mod­er­ate PPD group.

In Phase II, re­searchers re­port­ed a 12.2-point spread be­tween the drug and the place­bo, leav­ing Sage de­fend­ing a sig­nif­i­cant­ly re­duced mar­gin of im­prove­ment. And there were mixed re­sults on dura­bil­i­ty as well.

RBC’s Bri­an Abra­hams has pegged peak po­ten­tial sales at about $650 mil­lion, fac­tor­ing in a price of $15,000 to $20,000 per in­fu­sion — if it gets the green light. And an­a­lysts have shown con­sid­er­able en­thu­si­asm for an oral fol­low-up that could over­come the in­fu­sion hur­dle.

This new pri­or­i­ty re­view comes a day af­ter Loxo and Bay­er gained the same path­way for larotrec­tinib, the same day Astel­las bagged its own 6-month snap in­spec­tion for their AML drug. Reg­u­la­tors have been turn­ing to pri­or­i­ty re­views more fre­quent­ly as FDA com­mis­sion­er Scott Got­tlieb de­liv­ers on his promise to speed things up for drug de­vel­op­ers.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.

GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,300+ biopharma pros reading Endpoints daily — and it's free.